Clinical trial · Observational
Stem Cells in NF1 Patients With Tumors of the Central Nervous System
Development of Stem Cell Lines in Children With Neurofibromatosis Type 1 and Tumors of the Central Nervous System
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Suspended due to cessation of funding
Summary
Brief summary (as posted)
Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1. 2\. Develop isogenic NF1 wild-type (NF1+/+), NF1 heterozygous (NF1+/-) and NF1 homozygous (NF1-/-) iPSC lines from individual patients using CRISPR/CAS9 technology. 3\. Differentiate and characterize disease-relevant brain cells such as excitatory and inhibitory neurons, astrocytes and oligodendrocytes from patient-specific iPSC lines. 4\. Screen and identify the drug(s) that can reverse or alleviate the disease phenotypes.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neurofibromatosis Type 1 | — | UNRESOLVED | — |
| Tumors of the Central Nervous System | Central Nervous System Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Collection of Stem Cells | Diagnostic Test | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (1)
- measure
- The identity of mutations in NF1 genes will be measured.
- timeFrame
- June 2019
- description
- The stem-cell characteristics of patient-derived induced pluripotent stem cell (iPSC) lines will be measured and reported.
Secondary outcomes (3)
- measure
- The iPS cell lines with NF1 mutations will be engineered to inactivate the remaining NF1 wild-type or fix the mutant allele using CRISPR/CAS9 technology.
- timeFrame
- June 2019
- description
- The status of NF1 gene will be measured for the isogenic NF1 wild-type (NF1+/+), NF1 heterozygous (NF1+/-) and NF1 homozygous (NF1-/-) iPSC lines. The stem cell characteristics of isogenic NF1 iPSC lines will be measured.
- measure
- Measure neuronal characteristics of neurons derived from iPSC lines.
- timeFrame
- June 2019
- description
Eligibility
Eligibility (as posted)
- Sex
- All
Show eligibility criteria text
Inclusion Criteria: * Males or females of any age * Confirmed diagnosis of NF1 * Willingness to submit blood sample and collect clinical history * MRI documentation confirming tumor location in the central nervous system. * For study group d, "Non-NF1 full sibling for control purposes" subject must be a full sibling of a patient with confirmed diagnosis of NF1 and willing to submit blood sample and collect clinical history. Exclusion Criteria: * Does not have diagnosis of NF1 and CNS Tumor * Does not have full-sibling with NF1 and CNS Tumor diagnosis (for unaffected sibling cohort)
References
Publications (0)
Data not yet available